Dicerna Announces That Kyowa Hakko Kirin Has Selected a Second DsiRNA Therapeutic Candidate for Development, Triggering $5 Million Milestone Payment

Dicerna Announces That Kyowa Hakko Kirin Has Selected a Second DsiRNA Therapeutic Candidate for Development, Triggering $5 Million Milestone Payment
January 03, 2013

WATERTOWN, Mass.--(BUSINESS WIRE)--Dicerna Pharmaceuticals, Inc. (Dicerna), a RNA interference (RNAi) company focused on developing breakthrough oncology therapeutics, today announced that Kyowa Hakko Kirin Co., Ltd. (KHK) (TSE: 4151) has elected to advance the second therapeutic oncology candidate from the research to development stage. The achievement of this milestone triggers a $5 million payment from KHK to Dicerna.

Dicerna has a research collaboration and license agreement with KHK worth up to $1.4 billion for the research, development and commercialization of Dicer substrate siRNA (DsiRNA) pharmaceuticals and drug delivery systems for gene targets with therapeutic potential in oncology. In 2011, Dicerna announced that KHK had moved its first oncology candidate under the collaboration into development and also nominated a second oncology target. The milestone announced today is in regard to this second oncology target. DsiRNA delivered by KHK's drug delivery system to the target has yielded compelling activity in animal models of difficult-to-treat tumors.

"We are delighted that our collaboration with KHK has delivered a second product candidate that will advance to the development stage," said Douglas M. Fambrough, Ph.D., chief executive officer of Dicerna. "Along with Dicerna's own hepatocellular carcinoma program, we now have three announced oncology candidates, all utilizing Dicerna's Dicer Substrate Technology™, in development. Each of these is moving toward the clinic with the goal of addressing unmet cancer therapy needs."

"When we first entered this collaboration with Dicerna to access the Dicer Substrate Technology™, we saw the potential for RNAi technology to create novel therapeutics targeting key genes at the root of cancer," said Etsuo Ohshima, Ph.D., managing officer and vice president, head, research division at Kyowa Hakko Kirin. "This strategy is now providing KHK with multiple promising development candidates and helping to complement our oncology pipeline. The speed with which this second oncology program has moved from concept to development candidate is impressive. Highly active DsiRNAs combined with KHK's proprietary lipid nanoparticle delivery technology can be validated in vivo in less than a year."

"We have now reached a point where the platforms are sufficiently developed that there is a 'plug and play' reality to bringing therapeutic opportunities forward," said Bob Brown, Ph.D., chief scientific officer of Dicerna. "We will fully leverage this to create near-term product opportunities while pushing the technology to the next level."

About Dicer Substrate RNAi (DsiRNA)

Dicerna's DsiRNA molecules are uniquely structured and different from first generation siRNAs. Specifically, the company's DsiRNA molecules are 25 or more base pairs in length and are processed by the Dicer enzyme. The ability to engage the Dicer enzyme, which operates early in the gene silencing cascade, provides functional benefits over conventional, shorter siRNA molecules.

About Dicerna

Dicerna is a private, venture-backed biopharmaceutical company harnessing RNA interference (RNAi) to develop breakthrough, targeted cancer therapeutics. Utilizing its Dicer Substrate Technology™ platform and EnCore™ lipid nanoparticle delivery technology, Dicerna is developing a pipeline of programs to reach previously undruggable intracellular disease targets. The initial program Dicerna will bring to the clinic targets hepatocellular carcinoma, a disease of significant worldwide unmet need, through a DsiRNA delivered by an EnCore particle, directed to the MYC gene. The company has collaborations and major alliances with global pharmaceutical companies and will continue to build on this momentum to advance its research and development efforts. Founded in 2007, Dicerna is based in Watertown, Massachusetts. For more information, please visit www.dicerna.com.

About Kyowa Hakko Kirin

Kyowa Hakko Kirin is a leading biopharmaceutical company in Japan focusing on its core business area of oncology, nephrology and immunology/allergy.

Kyowa Hakko Kirin leverages antibody-related leading-edge technologies to discover and develop innovative new drugs aiming to become a global specialty pharmaceutical company which contributes to the health and well-being of people around the world. For more information, visit http://www.kyowa-kirin.com.


Dicerna Pharmaceuticals
Jim Weissman, 617-612-6214
Chief Business Officer
Pure Communications
Michele Rozen, 617-730-8284

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